Exploring Polycystic Kidney Disease Clinical Trials and New Treatments

This webinar will explore polycystic kidney disease (PKD) with a focus on autosomal dominant PKD (ADPKD), including clinical presentations, considerations for conducting clinical trials in this population and the future of ADPKD treatment.

In the United States, approximately 600,000 people have PKD¹, making it the fourth leading cause of kidney failure, and there is no cure presently available.²  Men and women are equally at risk for this inherited disorder, and about 90% of PKD cases are ADPKD, meaning only one copy of the abnormal gene, either PKD1 or PKD2, is needed to cause the disease.  Globally, ADPKD affects approximately 12 million people³ and is the most common cause of genetic kidney failure. Autosomal recessive PKD, or ARPKD, means that both parents must have and pass down the PKHD1 mutation to their child; ARPKD is extremely rare and only occurs in about one in 25,000 people.

Since 2014, genetic testing for ADPKD has improved in accuracy, availability and affordability, leading to higher usage; additional genes outside of PKD1 and PKD2 have also been associated with this disorder, and significant interest in improved outcomes for patients persists. There has been a noticeable increase in clinical trial activity related to potential new therapies for ADPKD over the last several years; tolvaptan was approved in 2015 and 2018 by Health Canada, the European Medicines Agency (EMA) and the US Food and Drug Administration, respectively, and there are more drugs in development for ADPKD now than ever before.⁴

The recently released Kidney Disease: Improving Global Outcome guidelines for ADPKD highlight the need for improved biomarkers of ADPKD progression and have led to focused research in the development of serum proteomics-based outcome prediction models.⁵ Beyond proteomics, another growing body of research surrounds ciliopathies, or primary cilia dysfunction.  As ciliopathies lead to a wide range of disorders, the most common of which is ADPKD, and primary cilia are critical for the regulation of cellular metabolism, glutamine utilization via asparagine synthetase (ASNS) is another potential area of interest as a target for therapy.

The featured speakers will highlight the current landscape for ADPKD, factors to consider for clinical trials and what the future may hold for possible new therapies. They will consider the novel challenges and opportunities that these evolving questions present in addressing the urgent need for increasing the number of safe and effective treatments for people living with ADPKD.

Register for this webinar to learn how polycystic kidney disease clinical trials are shaping the future of ADPKD treatment.

Speakers

Dr. Sayna Norouzi, Associate Professor of Medicine, Founder & Director of both the Polycystic Kidney Disease Clinic and the Glomerular Disease Clinic at Loma Linda University Medical Center

Dr. Sayna Norouzi, an Associate Professor of Medicine and Clinical Nephrologist at Loma Linda University Medical Center, finished her fellowship at Baylor College of Medicine in Houston, Texas. She is the Founder and Director of the Glomerular Diseases Clinics at Loma Linda and serves as the Co-Director of the online GlomCon Glomerular Diseases fellowship program. She is also the Founder and Director of the Polycystic Kidney Disease clinic. She has been recognized as the Educator of the Year for two consecutive years and is a member of the Alpha Omega Alpha Medical Honor Society. Dr. Norouzi focuses on glomerular diseases and polycystic kidney disease, actively participating in various research projects and clinical trials.

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Dr. Hannah Troutman, Nephrologist and Associate Medical Director at the PPD clinical research business of Thermo Fisher Scientific

Dr. Hannah R. Troutman is an Associate Medical Director within the division of Pharmacovigilance of the Clinical Research Group at the PPD clinical research business of Thermo Fisher Scientific. She is board-certified in Internal Medicine and Nephrology with more than 12 years of experience. She completed a residency in Internal Medicine at Pennsylvania Hospital, part of the University of Pennsylvania Health System. She completed a Nephrology fellowship with a concentration in transplantation at Medstar Washington Hospital Center/Georgetown Transplant Institute. Before joining the PPD clinical research business of Thermo Fisher Scientific, Hannah was in private practice and later in academic practice. She played an integral role in the development of the Crozer-Keystone Regional Kidney Transplant program. Later, as Medical Director with DaVita, she led the expansion of the home dialysis program and launched a Home Dialysis and transitional care unit at Thomas Jefferson University Health. There, she also served as a Co-Investigator for clinical trials. Hannah has served as a Clinical Educator and Assistant Professor at various academic institutions.

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(Moderator) Sarah Dixon Stump, MS, Project Oversight Senior Director at the PPD clinical research business of Thermo Fisher Scientific

Sarah Stump is a Senior Director within the PPD clinical research business of Thermo Fisher Scientific Cardiovascular and General Medicine (CVGM) group. She holds a Master’s degree in Community Health, with more than 19 years of experience in drug development, the CRO industry and academia. In her role as a Renal Indication Champion at the PPD clinical research business of Thermo Fisher Scientific, she has worked to expand connections with key renal patient advocacy groups and KOLs and has co-authored several publications and poster presentations.

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